小核酸药物行业深度研究报告：RNA精准医疗时代的崛起与挑战

Investment Rating - The industry rating is recommended for investment [5] Core Insights - The small nucleic acid drug industry is positioned as a core track in precision medicine due to technological breakthroughs and differentiated treatments, focusing on ASO and siRNA types to regulate genes at the mRNA level, overcoming traditional "undruggable" targets and covering thousands of rare and chronic disease-related targets [2][11][12] - The global market for small nucleic acid drugs is rapidly expanding, with a projected growth from $2.7 billion in 2019 to $5.7 billion in 2024 (CAGR of 16.2%), and expected to reach $20.6 billion by 2029 (CAGR of 29.4%), driven by the increase in chronic diseases and significant breakthroughs in liver-targeted technologies [3][21] - The industry is characterized by a dual structure of "international leaders dominating + local companies breaking through," with companies like Alnylam and Ionis leading the market while local firms focus on niche areas [3][40] Summary by Sections Industry Overview - Small nucleic acid drugs leverage unique mechanisms to achieve precise gene regulation, offering advantages over traditional small molecules and antibody drugs [11][12] - The technology has evolved from being considered "undruggable" to providing viable treatment options for various diseases, including rare genetic disorders [12][13] Market Expansion - The market is transitioning from rare diseases to common diseases, with chronic disease treatments expected to dominate in the future [21][22] - The introduction of significant products like Novartis' Inclisiran is anticipated to drive sales growth, with a projected revenue of $750 million in 2024 [21][22] Competitive Landscape - International leaders like Alnylam have established a strong market presence with multiple commercialized products, while local companies are innovating in specific niches [40][41] - Alnylam's revenue reached $2.62 billion in the first three quarters of 2025, marking a significant milestone in profitability and cash flow [40][41]